#United States #Pittsburgh #Ionis Pharmaceuticals #Tryngolza #PANTHERx Rare

PANTHERx® Rare and Its Commitment to Rare Disease Patients

In a significant step towards improving the lives of patients with rare diseases, PANTHERx® Rare has been chosen by Ionis Pharmaceuticals to manage the distribution of TRYNGOLZA™ (olezarsen). This groundbreaking treatment is the first FDA-approved drug aimed at reducing triglyceride levels in adults afflicted by familial chylomicronemia syndrome (FCS).

Understanding FCS and Its Implications

FCS is a genetic disorder that disrupts fat metabolism, leading to dangerously high triglyceride levels in the blood. These patients can experience acute pancreatitis and long-term health complications, making effective treatment crucial. TRYNGOLZA™ functions by degrading apoC-III mRNA, thus reducing the production of apoC-III, the key protein involved in triglyceride regulation. As a result, it presents a new hope for patients who previously had limited options.

The Role of PANTHERx® Rare

PANTHERx® Rare is recognized for its dedication to rare diseases and has developed extensive patient access services. This new partnership marks an exciting chapter in the pharmacy's mission to deliver and support innovative treatments for marginalized patient populations. Rob Snyder, Executive Chair of PANTHERx® Rare, emphasized the significance of this collaboration, stating that the streamlined access process would greatly benefit adults suffering from FCS. By easing the path from manufacturer to patient, PANTHERx® aims to improve patient well-being significantly.

A Dual-Accredited Specialty Pharmacy

Apart from facilitating the distribution of cutting-edge treatments like TRYNGOLZA™, PANTHERx® Rare holds a commendable place in the healthcare ecosystem as a dual-accredited specialty pharmacy. The organization has exceptional credentials from the Accreditation Commission for Health Care (ACHC) and the Utilization Review Accreditation Commission (URAC) for rare diseases. Furthermore, it has been honored with the MMIT Patient Choice Award multiple times, underscoring its commitment to patient satisfaction.

The Bigger Picture

Currently, there are more than 7,000 identified rare diseases affecting an estimated 25-30 million people across the United States, with over 90% lacking FDA-approved treatments. The increasing number of FDA orphan drug approvals highlights the evolving landscape of rare disease therapies, generating hope for patients and their families. Initiatives like those by PANTHERx® Rare play an essential role in bridging the gap between innovative treatments and patients in need.

Conclusion

This collaboration between PANTHERx® Rare and Ionis Pharmaceuticals signifies a major advancement for those battling FCS. As PANTHERx® Rare heads into this partnership, the commitment to delivering essential therapies and improving patient access is poised to make a profound impact on the rare disease community, offering renewed hope for a life with better health outcomes.